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Taking Science Personally

A few years after Michael J. Fox revealed that he had Parkinson’s disease, he attended the annual charity auction for the Robin Hood Foundation, a New York philanthropy financed largely from donations by wealthy hedge fund managers. Mr. Fox was in his late 30s at the time. He had been living with Parkinson’s since 1991, when he received the diagnosis at the age of 30, but had kept quiet about it while he continued his acting career. It was only in 1998, when his symptoms — the uncontrollable tremors and halting speech that characterize those with Parkinson’s — had become pronounced enough that he could no longer continue with his hit show “Spin City” that he finally let the news out.

Because he is a well-known, and well-liked, actor, there was naturally an outpouring of support from people all around the world — “much more attention that I expected,” Mr. Fox recalled recently. Although he recoiled at the thought of becoming “the P.D. poster boy,” as he later put it, he also realized that his celebrity gave him a currency that might be useful in helping bring about a cure for Parkinson’s disease. Early on, he testified before Congress about the lack of federal funds going to Parkinson’s research. Beyond that, though, he wasn’t sure what he could do.

That evening at the Robin Hood auction, as he watched hedge fund managers trying to outdo themselves in bidding for charity items, it occurred to him. “You could see at that dinner that you could definitely get stuff funded,” he said. “I woke up to the fact that if I came up with a plan, I could have access to people.”

Mr. Fox did indeed come up with a plan. Like many celebrities who embrace a cause, he started a foundation, the Michael J. Fox Foundation. Last year, it spent $25.2 million fighting Parkinson’s disease. Although he does not run the foundation — never has, in fact — he serves as its chairman and inspirational leader.

There are thousands of foundations around the world dedicated to eliminating one disease or another; the founding of yet another isn’t exactly news. Collectively, they spend hundreds of millions of dollars each year. And yet — and here is the rub — the diseases they are trying to cure remain stubbornly uncured.

What makes the story of the Michael J. Fox Foundation different — nay, what makes it important — is that it doesn’t just dole out money to scientists and hope for the best. It has used its money to take control of Parkinson’s research like few other foundations have ever done. In the process of trying to solve the mysteries of Parkinson’s, it has upended the way scientific research is done, and the way academics interact with pharmaceutical and biotech companies, at least in its little corner of the world. It demands accountability and information sharing that is almost unheard of in the broad scientific community. And it has managed to become, in its short seven-year life, the most credible voice on Parkinson’s research in the world. “If you are a serious Parkinson’s researcher,” said Malu Tansey, a scientist at the University of Texas Southwestern Medical Center, “you live and die by the Fox Foundation’s Web site.”

In this, it has joined a small but growing group of what might be called activist disease foundations — foundations that operate with speed and urgency and a business model completely unlike the traditional foundation model, or the National Institutes of Health, for that matter. The Cystic Fibrosis Foundation was one of the instigators of this movement, more than a decade ago, as was Michael Milken’s Prostate Cancer Foundation, formerly CapCure. The Myelin Repair Foundation, for multiple sclerosis, and the Multiple Myeloma Research Foundation are also members of this tribe. They all use their money, unabashedly, as weaponry, to finance targeted research, hold conferences where scientists must share information and underwrite clinical trials conducted by drug companies. They are hard not to admire.

Yet Cystic fibrosis is still with us, as is prostate cancer, multiple sclerosis and multiple myeloma. And so is Parkinson’s disease. Which leads to an obvious question: after seven years, has enough time passed to ask whether this new business model is really any better than the old one?

“SEVEN years,” groused Andrew S. Grove when I visited him recently in Silicon Valley. “And what do we have to show for it? Nothing.” He spat out the last word with a classic Grove-like flourish of disgust.

The former chairman and chief executive of Intel, Mr. Grove, now 71, also has Parkinson’s disease — as well as a small, seven-year-old foundation, the Grove Foundation, trying to combat it. In addition, Mr. Grove plays an important but ad hoc advisory role for the Michael J. Fox Foundation, to which he has made a $40 million bequest.

It is clear that the formation of these two foundations in 2000 marked the beginning of targeted Parkinson’s research. Before then, what existed was broader university research into both the brain and the nervous system, some of which was applicable to Parkinson’s disease but much of which was not. Much of it was financed by the National Institutes of Health, an agency of the Department of Health and Human Services. As for industry, because there are far more Alzheimer’s patients than Parkinson’s patients, much of the cutting-edge industry neuroscience was designed for the former group rather than the latter. When Mr. Fox started his foundation, he was filled with optimism, and he grandly announced that his organization wanted to find a cure in 10 years. For that matter, Mr. Grove was pretty optimistic, too: when he started his foundation, he felt sure that by backing certain kinds of stem cell research (work the government was refusing to finance), he could quicken the move toward a cure. At the time, there was a widespread belief in the Parkinson’s community that stem cell research would lead to a cure.

But both expectations turned out to be unrealistic; stem cells, though they still hold promise, have not turned out to be the Parkinson’s silver bullet that scientists once hoped they might be. In the last few years, Parkinson’s research has moved in other, more promising directions. Similarly, Mr. Fox and his colleagues at the Fox Foundation have come to realize that finding a cure for Parkinson’s is slow, painstaking work and that to do it in 10 years is wildly unrealistic.

Which is really what Mr. Grove was referring to with his dour remark to me. “The most important thing we have figured out is the various things we didn’t know and should have known,” he said. “We figured out we are further back than we thought. That is what we have to show for seven years. It is very unsatisfactory.”

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STAR POWER From left, Sean Twitty, Ben Stiller, Michael J. Fox, Marc Lasry and Derek Jeter at a Fox foundation fund-raising dinner on Wednesday in Manhattan.Credit
Jennifer S. Altman for The New York Times

Which, in fact, is something — you have to be doing targeted research even to discover what you need to discover. And in truth, Mr. Grove isn’t quite as pessimistic as he appeared on the day I visited him. He has since peppered me with speeches he has made and slide shows he has presented that show him to be a curmudgeon with a purpose. He sees his role as being someone whose job it is to push and prod and not let anyone become satisfied with the progress they have made. Including, as it turns out, the Michael J. Fox Foundation.

Mr. Fox, a man of keen intelligence who nonetheless likes to describe himself as a high school dropout, did not have a clear idea at the beginning how his foundation would operate. But he did have some overarching principles. He wanted the foundation to be run by someone from the business world, rather than a career philanthropist. (“My hedge fund friends kept telling me what I was doing sounded like a start-up,” he recalls.) He met Deborah Brooks, who had spent the early part of her career at Goldman Sachs, and hired her pretty much on the spot to be the first chief executive. (She is currently a Fox Foundation fund-raiser.)

He wanted the foundation to have a powerful sense of urgency, and for that reason he insisted that it had to spend the money it took in, rather than building up a large endowment, which often becomes the end instead of the means. (“The goal is to go out of business,” he said.) He wanted it to be an organization that was about science rather than, say, patient advocacy. Mainly, he wanted his foundation to approach the problem differently from the way other foundations had approached it. But he didn’t know exactly what that meant.

Katie Hood, the current chief executive, recalls that when she first joined the organization about a year and a half into its existence, it saw its primary mission as “more money faster.” That is, it awarded scientific grant money faster than the N.I.H. — or other philanthropies, for that matter — but in other ways its model wasn’t all that different. It had a scientific advisory board that met several times a year — which meant that important decisions had to be deferred until they convened.

The N.I.H. had lengthy grant application forms and gave money for five years. The Fox Foundation’s forms were shorter, and it gave money for two years. The Fox Foundation’s approach was streamlined and took more risk, to be sure. That said, it wasn’t radically different from the government’s general tack.

Ms. Brooks recalls a critical, early meeting in New York attended by Mr. Grove. The Fox and Grove foundations had put together a partnership to finance stem cell Parkinson’s research, and for three hours Mr. Grove listened carefully as the Fox Foundation’s staff laid out all of its activities. When they had finished, Mr. Grove said, “Congratulations, Debi. You look just like the N.I.H.” It was not meant as a compliment.

What is wrong with N.I.H. funding? To most people, it is a model that makes perfect sense — in part because it is what we are used to. The government accepts grant applications from scientists, and then metes out money to a handful of those with ideas that it deems most promising.

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ON THE CASE Andrew S. Grove, former chief executive of Intel, started the Grove Foundation to combat Parkinsons disease.Credit
Jim Wilson/The New York Times

But from the point of view of a disease foundation desperate to find a cure, everything is wrong with it. The government grants tend to go to low-risk projects, so the kind of science that leads to big breakthroughs tends to go underfinanced. The N.I.H. pays for areas of science that are high profile or have a large constituency. It has no connection to industry research, so it is largely through happenstance that government-financed research turns into medicine that helps make us better. And once the money is channeled, that’s pretty much the end of it: there is no follow-up. People like Mr. Grove believe that there is nothing urgent about N.I.H. money — and nothing targeted.

The N.I.H. disputes some of this. “We at N.I.H. share Andy Grove’s sense of urgency to find cures for Parkinson’s disease and other devastating disorders,” said Dr. Robert Finkelstein, director of extramural research at the National Institute of Neurological Disorders and Stroke. He added that there were many projects that “involve interactions with industry, and all of them have concrete benchmarks.”

Meanwhile, the Fox Foundation wanted a change: in how it financed research, and what kind of research it financed. Instead of simply doling out money for a certain period of time — and hoping for the best — it began to keep closer track of researchers, using its money to accelerate research that was going well, or shift direction if it wasn’t. It expected its researchers to report problems and progress. It established markers and other goals that researchers were expected to meet. It insisted that the money it gave went very specifically to research that was directly related to Parkinson’s — and it was unafraid to finance risky ideas. “We throw 100 ideas up against the wall,” Ms. Brooks said. “One of the most important things we do is de-risk ideas” — and in so doing make them more palatable for industry and other researchers to pursue.

The Fox Foundation began to describe the research it financed as “translational” — meaning the applied biology research it paid for was intended to eventually translate into a treatment or a drug. Academic scientists don’t usually think in those terms. But those were the terms required to get Michael J. Fox money. It was using its money to change academic behavior. Suddenly, a younger generation of researchers — people who did not yet have traditional, established career paths — became very interested in doing things the Michael J. Fox way. They were perfectly happy to meet goals and share information. They understood that the ultimate goal was to find a cure for Parkinson’s disease, and they were fine with that. It was a way to get important science financed.

A few years later, Ms. Brooks and Mr. Fox made another important shift. Instead of relying on the scientific advisory board to make “yea” or “nay” decisions on grants, it decided to hire its own Ph.D.’s. It now has six in-house scientists. The Ph.D.’s allow the foundation to move faster still, since it no longer has to wait for the scientific advisory board to meet to make decisions. But it has other benefits as well: the Fox scientists are young, early enough in their careers that they are not set in their ways. There are perfectly happy to ask different kinds of questions and do things in a different way. In effect, they have allowed the Michael J. Fox Foundation to not just pursue important research already being done, but also to set the agenda for research it thinks ought to be pursued. And that is what has happened.

Another piece of the puzzle has to do with working with biotech and pharmaceutical companies. This might seem like something obvious for disease-oriented philanthropies to do, but it’s not. When the Michael J. Fox Foundation held its first meeting with industry representatives in 2004 — mainly to find out what it could do to push Parkinson’s research along within the industry — it was the first such meeting just about anybody in the biotech industry had ever attended. The point was to find out what the industry wanted and needed if companies were going to conduct clinical trials for drugs that might help Parkinson’s patients.

One thing the Fox Foundation discovered was that industry research and academic research were very different. Industry cared about “target validation,” for instance — affirming the impact of a molecule on a disease — which was a low priority for most academic researchers. It also began financing partnerships with industry. And so back to the question that began this article: Is it making a difference?

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MORE MONEY FASTER Katie Hood of the Fox foundation said her group could make science grants faster than the N.I.H.Credit
Jennifer S. Altman for The New York Times

As Mr. Fox himself has discovered, seven years turns out to be a not-very-long time to be fighting a disease whose root cause is still not yet known. “It is a tremendously idiosyncratic disease,” he said. “The level of research that is going on is still pretty basic. We are not just looking for a cure, we are looking for a cause, and different ways to deal with the side effects of the medication.”

But there is also no doubt that far more progress has been made than would be the case if the Michael J. Fox Foundation didn’t exist. A few months ago, I attended another of the foundation’s regular meetings of industry representatives and academic researchers. It was held in a Chicago hotel, and the meeting room was packed. Scientist after scientist made presentations — and were then peppered with questions from industry representatives. The Fox representatives took furious notes.

At this point, two drugs are in Phase II clinical trials thanks to Fox Foundation grants. And the Fox Foundation isn’t the only one having success; the Cystic Fibrosis Foundation’s efforts have gotten a drug to Phase III, the final phase before consideration by the Food and Drug Administration.

SO yes, progress. Not as fast as Mr. Fox — or anyone else connected with the foundation — would like, but more than enough, it seems to me, to validate their business model. “I learned that for this to be an effective organization, it really does have to be run like a business,” said Mr. Fox. “You can’t be afraid of making mistakes, but you have to have a certain level of efficiency. You need machinery to be in place. And you have to have a bottom line of some sort.”

As he spoke, in his office on the Upper East Side of Manhattan, his legs began twitching and shaking. Several times his foot hit a glass coffee table in front of him. His medication was starting to wear off, he explained with a small smile.

I asked him whether part of his motivation was to see himself cured someday. No, he said, that had never been his goal. “I think self-interest is a great starting point,” he replied, “but I don’t want it to cloud my thinking, and I don’t want the foundation to ever do anything with me in mind. This is a responsibility we have now. I want people with Parkinson’s to wake up knowing that there is someone trying to get this done. We want to be accountable to ourselves.”

He continued: “I have this image of bursting a door open and watching people go through it. And then, at some point, putting my own coat on and walking through the door.”

It won’t be in 10 years, that’s for sure. But the door is far more likely to swing open with the Michael J. Fox Foundation than without it.